Legend Biotech Conference Spotlights CARVYKTI’s Curative Potential, Earlier Use

Key Points

• Legend Biotech used its investor event to argue that CARVYKTI has broad curative potential in multiple myeloma, with doctors citing long-term follow-up data showing some patients remain progression-free and treatment-free for more than five years after a single infusion.
• Company executives said CARVYKTI has become a major commercial success, generating nearly $2 billion in 2025 sales and treating more than 10,000 patients, while Legend expects to reach adjusted net income profitability this year.
• Physicians at the event said outcomes appear better when CARVYKTI is used earlier in the disease course and after effective bridging therapy, with lower toxicity and stronger progression-free survival than in later-line, higher-burden patients.

Legend Biotech NASDAQ: LEGN used an investor and analyst event to highlight long-term data and physician perspectives supporting broader use of CARVYKTI, its BCMA-directed CAR T-cell therapy for multiple myeloma developed, manufactured and co-promoted with Johnson & Johnson.

Chief Executive Officer Ying Huang said Legend is the “world’s largest standalone cell therapy company” and described CARVYKTI as the “fastest commercial launch among all CAR T therapies to date.” Huang said the product generated nearly $2 billion in total sales in 2025 and has treated more than 10,000 patients to date. She also said Legend had $835 million in cash and liquidity at the end of the first quarter and expects to achieve corporate profitability this year on an adjusted net income basis.

The event focused on CARVYKTI’s long-term efficacy, the case for earlier use in multiple myeloma and evolving safety management practices. Huang said the company views CARVYKTI as a one-time infusion that can be used in inpatient or outpatient settings and may offer lifetime cost savings for patients, healthcare practitioners and payers.

Physicians Highlight Long-Term Data and “Curative Potential”

Dr. Binod Dhakal, associate professor of medicine at the Medical College of Wisconsin, said CARVYKTI has changed how physicians discuss multiple myeloma, historically considered an incurable cancer.

Dhakal pointed to a proposed International Myeloma Working Group definition of cure in multiple myeloma, which includes sustained treatment-free minimal residual disease, or MRD, negativity for five years, sustained complete response, repeated MRD assessments and no evidence of disease by imaging.

Discussing long-term CARTITUDE-1 follow-up, Dhakal said that among 97 treated patients, about one-third were treatment-free and progression-free for more than five years after a single infusion. He also said 12 patients at one center with annual MRD and PET assessments were MRD negative and PET negative for five years, meeting the proposed definition of cure.

Dhakal said median overall survival in CARTITUDE-1 was about 61 months in a heavily pretreated population with a median of six prior lines of therapy. He contrasted that with historical expectations for similar triple-class exposed patients, saying they would have had median overall survival of less than a year.

Doctors Say Earlier CAR T Use May Improve Outcomes

Dr. Doris Hansen, assistant member of blood and marrow transplant and cellular immunotherapy at Moffitt Cancer Center, argued that earlier treatment with CARVYKTI is preferable because multiple myeloma becomes more difficult to treat and more genomically complex with each line of therapy.

Hansen said patients are lost at each treatment line, with attrition rates between 10% and 30% per line of therapy. She cited CARTITUDE-1 and CARTITUDE-4 data, including median progression-free survival of about three years in heavily pretreated CARTITUDE-1 patients and median progression-free survival as high as 50 months in certain triple-class exposed patients with three prior lines of therapy. In CARTITUDE-4, she said median progression-free survival had not been reached at 30 months in patients who were proteasome inhibitor and IMiD exposed and lenalidomide refractory.

Hansen also said safety appears to improve when CARVYKTI is used earlier. She noted that Parkinsonism was reported in 6% of patients in CARTITUDE-1 at five years, while CARTITUDE-4 had one patient with Parkinsonian features, or less than 1%. She added that manufacturing success was 99% when CAR T was moved earlier in the disease course.

In the question-and-answer session, Hansen said she is treating all eligible patients in second line with CARVYKTI, whether they are functional high risk or standard risk, unless logistical or comorbidity issues prevent it. Dhakal and Dr. Surbhi Sidana, associate professor of medicine at Stanford University, described similar practice patterns, saying they recommend CAR T as early as first relapse for eligible patients.

Bridging Therapy Emphasized as Safety Strategy

Sidana focused on safety management and said disease burden before CARVYKTI treatment is an important factor. She said patients who did not respond well to bridging therapy had worse progression-free survival and overall survival, and that going into CARVYKTI with high disease burden is “not a good idea.”

Sidana cited multiple data sets, including CARTITUDE-4 and real-world consortium data, to argue that CARVYKTI should be used as a consolidative therapy after effective disease debulking. In a U.S. Multiple Myeloma CAR T Consortium data set of more than 750 patients, she said 21 of 22 Parkinsonism cases occurred in patients who had not responded to bridging therapy. She said the risk of Parkinsonism was 5% in non-responders compared with 0.5% in patients with a partial response or better to bridging therapy.

Sidana said manufacturing constraints are “a non-issue” at this point and that bridging options depend on the patient’s prior treatments and refractory status. She said talquetamab is often used in more refractory patients, while earlier-line patients may receive other options such as daratumumab-based bridging if appropriate.

CAR T Versus Bispecifics and Future Treatment Settings

The panel also discussed sequencing CARVYKTI with bispecific antibodies. Dhakal said that for eligible patients, his preference is to offer CAR T first, citing CARVYKTI’s potential curative option, one-time treatment and quality-of-life considerations. Hansen said Immunotherapy Working Group guidance recommends CAR before bispecific therapy for eligible patients.

A company representative also discussed two real-world data sets, including a German registry of 606 patients and a U.S. TriNetX analysis of 389 patients. The representative said both supported the idea that earlier CARVYKTI use and CAR T before bispecific therapy were associated with better outcomes, while cautioning that the TriNetX analysis was not randomized and could not perfectly match patient characteristics.

Panelists said they are awaiting data from ongoing frontline studies, including CARTITUDE-5 in transplant-ineligible or transplant-delayed patients and CARTITUDE-6 comparing CARVYKTI head-to-head against transplant in transplant-eligible patients. Sidana said the field is moving toward using the most effective therapies earlier, but added that physicians need to wait for the data.

Asked about in vivo CAR T approaches, Hansen, Sidana and Dhakal described the technology as exciting but early. Hansen said there is limited follow-up and more information is needed on safety and durability. Huang declined to provide details on Legend’s own in vivo program ahead of a medical meeting abstract publication.

Legend also said demand is shifting earlier. A company representative said that, on the most recent earnings call, the company reported 41% of CARVYKTI patients were coming from second and third line treatment settings. The representative said Legend is expanding education, treatment-center access and patient activation efforts, including targeted direct-to-consumer advertising and outreach through social media.

About Legend Biotech NASDAQ: LEGN

Legend Biotech NASDAQ: LEGN is a commercial-stage biopharmaceutical company specializing in the development and commercialization of chimeric antigen receptor T-cell (CAR-T) therapies for oncology. Headquartered in Somerset, New Jersey, with research and development operations in Shanghai, the company leverages a global infrastructure to advance innovative cellular therapies. Legend Biotech pursues a strategy of strategic collaboration to extend its reach, most notably through its partnership with Janssen Biotech, a subsidiary of Johnson & Johnson.

The company's lead asset, ciltacabtagene autoleucel (commercially marketed as Carvykti), is a B-cell maturation antigen (BCMA)–directed CAR-T therapy for the treatment of relapsed or refractory multiple myeloma.

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